Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome

What Was Tested

The trial (phase 2) is built around Oxytocin for hyperparathyroidism.

This study is a phase 2 randomized double blind 8-week treatment trial of intranasal OXT vs. placebo in 50 subjects aged 5 to 17 years with PWS in order to assess IN-OXT's affect on measurements of (1) eating behaviors (2) repetitive behaviors (3) weight and body composition (4) quality of life (5) salivary OXT and hormone levels (including ghrelin, pancreatic polypeptide, peptide YY, Glucagon-Like Peptide-1 (GLP-1), insulin, glucagon, testosterone, and estrogen).

It had two arms (groups): Experimental: Intranasal Oxytocin (IN-OXT) (experimental) and Placebo Comparator: Matched Placebo (placebo comparator).

The registry classes the interventions as drug.

More on the Plan

Prader-Willi Syndrome (PWS) is a rare neurodevelopmental disorder caused by lack of expression of paternally derived imprinted material on chromosome 15q11-q13.

PWS is characterized by mild to moderate intellectual disabilities, repetitive/compulsive behaviors and rigidity, social cognition deficits and severe hypotonia at birth, followed by the onset of hyperphagia later in life.

Oxytocin has been implicated in the pathophysiology of PWS and there have been small studies of intranasal oxytocin (IN-OXT) in this population.

STUDY DESIGN: This is an 8-week double-blind, randomized study in 50 children with PWS aged 5-17.

Who Took Part

Enrollment: 32 actual participants took part.

It was open to people of any sex aged from 5 years up to 17 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Male or female pediatric outpatients aged 5 to 17 years. Must be in PWS nutritional phase 2b or 3 as determined by PI. Diagnosis of PWS confirmed by patient medical records. A score of at least moderate severity on the Hyperphagia Questionnaire for Clinical Trials at both screening and baseline visits. Stable dosages of hormone treatments (including testosterone and estrogen supplements) for 4 weeks prior to randomization and for the duration of the study.

The full record lists 9 inclusion and 13 exclusion criteria; among the exclusions: exposure to any investigational agent in the 30 days prior to randomization; child not receiving growth hormone treatment; children weighing less than 40 lbs.

How the Study Was Run

Design: randomized, parallel-group and quadruple-blind.

Because people in this Oxytocin study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

Groups ran in parallel: each of the hyperparathyroidism participants stayed in one arm throughout.

Blinding on both sides kept the hyperparathyroidism participants and researchers unaware of who received Oxytocin.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2018-04-11 with completion listed for 2022-08-29.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

It also tracked 13 secondary outcomes, for example Change in Repetitive Behavior, Change in Rigid Behavior, Change in Body Weight, Body Composition, Change in Body Mass Index (BMI) and Change in Caregiver Strain.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Oxytocin in 32 people with hyperparathyroidism.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Oxytocin compound page, Hyperparathyroidism research.